Technology | 2022.05.19
Neurogene’s Gene Replacement Program is the Eighth Biopharmaceutical Company Program Facilitated by the Rett Syndrome Research Trust
The research and resource development efforts of the Rett Syndrome Research Trust (RSRT) have helped generate an eighth biopharmaceutical program that tackles the root cause of Rett syndrome. Neurogene, a company founded on the vision to push the boundaries of genetic medicine to address complex and devastating neurological diseases, announced that it will be expanding the company’s development pipeline with the addition of NGN-401 for Rett syndrome. NGN-401 is a gene replacement approach that utilizes Neurogene’s novel EXACT gene regulation technology. Stuart Cobb, PhD, Chief Scientific Officer at Neurogene and Simons Fellow at the University of Edinburgh, presented preclinical efficacy and safety data for NGN-401 at the 25th Annual Meeting of the American Society for Gene and Cell Therapy. Gene replacement is one of six strategies championed by RSRT to target the root cause of Rett syndrome and is the strategy closest to the clinic.
(PRWeb May 19, 2022)
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